A closure of a revolutionary British project developing a treatment for cystic fibrosis was imminent. But thanks to renewed funding, now the project is saved said Professor Eric Alton, the leader of the project.
The team of scientists based in Edinburgh, Oxford and London were developing a treatment which involves putting genes into the lungs of patients suffering the fatal, inherited wasting disease, passed early clinical trials in 2010. But the recession badly affected the Cystic Fibrosis Trust, the charity that had funded the programme, and work had to stop last year just as the consortium was closing in on its goal.
The plight of the scientists and their patients was highlighted in a series of articles published in the Observer, and last week the Medical Research Council and the National Institute for Health Research announced they had said that they would provide more than £4m to allow the project to continue. “This funding will demonstrate how science in the lab can be translated into gene therapy for patients,” said the health secretary Andrew Lansley.
Cystic fibrosis affects 9,000 people in Britain and is caused by a mutant gene that prevents cells from producing healthy digestive juices, sweat and mucus. Bodily fluids become thick and sticky and clog up lungs and digestive tracts, which then become infected. Around 150 babies a year are born with the disease in Britain. The development of antibiotics has helped to keep them alive, but even today few live beyond their late 30s. Patients survive only by going through long daily physiotherapy sessions, the consumption of dozens of vitamin and digestive enzyme tablets, and the constant use of antibiotics and asthma inhalers. The cause of CF, mutations in a gene located on chromosome 7, was identified in 1989, opening the door to replacing this faulty gene using gene therapy.
Scientists at the Cystic Fibrosis Gene Therapy Consortium have isolated the healthy version of the gene and coated it in a fatty chemical known as a liposome. Patients inhale droplets of these liposome-coated genes, which should be taken up by cells in their lungs to replace their faulty genes. The groundbreaking gene therapy trial for cystic fibrosis (CF) will begin in March. One hundred and thirty adults and children with CF will take part in the largest trial of its type yet, coordinated by the UK Cystic Fibrosis Gene Therapy Consortium (GTC).
The second, lab-based study will investigate a more advanced version of the therapy using a modified virus to carry the replacement gene into the lungs, which could in future lead to a more efficient delivery mechanism.
Professor Eric Alton, the GTC Coordinator, from Imperial College London and consultant physician at Royal Brompton Hospital said, “Conventional treatments have extended the life expectancy for people with CF. We’re hoping that this therapy will achieve a step change in the treatment of CF that focuses on the basic defect rather than just addressing the symptoms. This trial will assess if giving gene therapy repeatedly for a year will lead to the patients’ lungs getting better. Eventually we hope gene therapy will push CF patients towards a normal life expectancy and improve their quality of life significantly. We would like to thank the NIHR and MRC for their confidence in us, and also all of those whose support has reinforced our determination to carry out this programme for the benefit of CF patients. We would also like to thank the Cystic Fibrosis Trust and the many families and supporters for funding our programme up to this stage.”
The outcome of the trial will be known in spring 2014 and regular progress reports will be posted on the Consortium’s website.
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